Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates.

ABSTRACT: HSC transplantation using genetically modified autologous cells is a promising therapeutic strategy for various genetic diseases, cancer, and HIV. However, for many of these conditions, the current efficiency of gene transfer to HSCs is not sufficient for clinical use. The ability to increase the percentage of gene-modified cells following transplantation is critical to overcoming this obstacle. In vivo selection with mutant methylguanine methyltransferase (MGMTP140K) has been proposed to overcome low gene transfer efficiency to HSCs. Previous studies have shown efficient in vivo selection in mice and dogs but only transient selection in primates. Here, we report efficient and stable MGMTP140K-mediated multilineage selection in both macaque and baboon nonhuman primate models. Treatment con

SUBMITTER: Beard BC 

PROVIDER: S-EPMC2898586 | biostudies-literature | 2010 Jul

REPOSITORIES: biostudies-literature